Clinical efficacy and safety of Octocog alfa in Chinese patients with hemophilia A: One-year follow-up results from the Antihemophilic Factor Hemophilia A Outcome Database (AHEAD) study
10.3760/cma.j.cn121090-20241022-00410
- VernacularTitle:Octocog alfa在中国血友病A患者中的临床疗效和安全性：AHEAD研究1年随访结果
- Author:
Runhui WU
1
;
Zhenyu LI
;
Jing SUN
;
Xin DU
;
Xinsheng ZHANG
;
Ying WANG
;
Qun HU
;
Rongfu ZHOU
;
Joan GU
;
Randy GUERRA
;
Renchi YANG
Author Information
1. 首都医科大学附属北京儿童医院，北京 100045
- Publication Type:Journal Article
- Keywords:
Recombinant human coagulation factor Ⅷ;
Hemophilia A;
Clinical efficacy;
1-year follow-up
- From:
Chinese Journal of Hematology
2025;46(8):705-712
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To evaluate the long-term efficacy and safety of recombinant coagulation factor Ⅷ (Octocog alfa) in Chinese patients with hemophilia A (HA) enrolled in the International Antihemophilic Factor Hemophilia A Outcome Database (AHEAD) study (NCT02078427) .Methods:Enrollment of Chinese patients in the AHEAD study was completed by January 2021, and data were collected up to July 15, 2022. This study primarily assessed patients in terms of the Gilbert score, global gait score within the Hemophilia Joint Health Score (HJHS), annualized bleeding rate (ABR), annualized joint bleeding rate, and adverse events.Results:A total of 168 male patients were included in this study, of which 113 received prophylactic treatment and 53 received on-demand treatment. The average age of the patients was 21.4±13.37 years. Compared with baseline, the global gait score within HJHS significantly decreased during the 1-year follow-up in patients with moderately severe HA in the prophylactic treatment group ( P=0.01) and on-demand treatment group ( P=0.008). The mean reduction in Gilbert score was greater in the prophylactic treatment group than in the on-demand treatment group (28.6% vs 8.2%). The average ABR decreased significantly during the 1-year follow-up (3.70 vs 7.78, P=0.01) in the prophylactic treatment group, particularly in patients with severe HA (2.14 vs 8.98, P=0.006) and pediatric patients (2.1 vs 4.73, P=0.03). The ABR score also decreased significantly in the moderate-dose prophylactic treatment group ( P=0.015). During the 1-year follow-up, 25 patients (14.9%) reported 39 adverse events, with only one patient developing treatment-related F Ⅷ inhibitor. Conclusion:Joint mobility improved in patients receiving either prophylactic or on-demand Octocog alfa. Bleeding episodes significantly reduced in patients receiving prophylactic treatment, particularly in pediatric patients and those with severe HA.
